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Neuromuscular Disorders Clinical Trials

Repoce: A Phase 3, Multicenter, Efficacy and Safety of Pozelimab and Cemdisiran Combinations Therapy in Patients with Symptomatic Generalized Myasthenia Gravis

Aims: The primary purpose of this research study is to evaluate the effect of pozelimab and cemdisiran on daily functioning that is impacted by signs and symptoms in patients with symptomatic generalized myasthenia gravis.
Principal Investigator: Alexandru Barboi, MD, FACP
NorthShore Project Number: EH21-282
Contact: Please call 847.503.4333 with questions regarding the study
Open to Enrollment: Yes

AMULET: Interventional, randomized, double-blind, parallel-group, placebo-controlled, multi-center study to assess the efficacy, safety and tolerability of Lu AF82422 in patients with Multiple System Atrophy

Aims: Multiple System Atrophy (MSA) is a rare degenerative disorder affecting balance, movement and the body’s involuntary (autonomic) functions, including blood pressure, breathing and bladder function. The purpose of this study is to find out whether a potential new treatment, the investigational drug Lu AF82422, is safe and effective in people living with MSA. This study will compare the effects of the investigational drug Lu AF82422 to placebo (inactive medication).
Principal Investigator: Alexandru Barboi, MD, FACP
NorthShore Project Number: EH21-278
Contact: Please call 847.503.4333 with questions regarding the study
Open to Enrollment: Yes

ARGNX-117-2002: A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, Multi-Center Trial to Evaluate the Efficacy, Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 2 Dose Regimens of ARGX-117 in Adults With Multifocal Motor Neuropathy

Aims: This phase 2 clinical trial serves to evaluate the safety and efficacy of 2 dose regimens of ARGX-117 versus placebo, in participants with Multifocal motor neuropathy (MMN) previously stabilized with IVIg. MMN is a rare neuropathy characterized by progressive asymmetric weakness and atrophy without sensory abnormalities. The objectives of the study include evaluating the safety and tolerability of ARGX-117 compared to placebo in adult participants previously stabilized with IVIg and to evaluate the efficacy of ARGX-117 compared to placebo on muscle strength and/or motor function in adult participants previously stabilized with IVIg.
Principal Investigator: Alexandru Barboi, MD, FACP
NorthShore Project Number: EH22-033
Contact: Please call 847.503.4333 with questions regarding the study
Open to Enrollment: Yes

RESTORE: A Clinical Study of Patients with Symptomatic Neurogenic Orthostatic Hypotension to Assess Sustained Effects of Droxidopa Therapy

Aims: The purpose of this study is to evaluate the durability, effectiveness and safety of the study drug, droxidopa, in those with neurogenic orthostatic hypotension (NOH). Droxidopa (NORTHERA) has been approved in the United States for treatment of NOH. However, effectiveness beyond 2 weeks has not been demonstrated. This study is placebo-controlled to assess the benefits beyond 2 weeks and is therefore investigational. Total study participation can be up to 36 weeks.
Principal Investigator: Alexandru Barboi, MD, FACP
NorthShore Project Number: EH18-106
Contact: Please call 847.503.4333 with questions regarding the study
Open to Enrollment: No

RAISE XT: A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis

Aims: The purpose of this research study is to provide access to Zilucoplan for subjects with Generalized Myasthenia Gravis (gMG) who have completed a qualifying Ra Pharmaceuticals sponsored Zilucoplan study and who wish to continue receiving Zilucoplan. This study will also evaluate the long-term efficacy of Zilucoplan in subjects with gMG who have completed the qualifying Ra Pharmaceuticals sponsored Zilucoplan study.
Principal Investigator: Alexandru Barboi, MD, FACP
NorthShore Project Number: EH21-143
Contact: Please call 847.503.4333 with questions regarding the study
Open to Enrollment: No

Synuclein-One Study
Aims: the purpose of this research study is to determine whether small skin biopsies can be used as a novel method of detecting diseases collectively known as synucleinopathies which include Parkinson disease (PD), Multiple System Atrophy (MSA), Dementia with Lewy bodies and Pure Autonomic Failure (PAF). This study will evaluate the effectiveness of this skin biopsy test to confirm a diagnosis of a synucleinopathy and ensure that testing abnormalities are not detected in healthy individuals.
Principal Investigator: Alexandru Barboi, MD, FACP
NorthShore Project Number: EH21-081
Contact: Please call 847.503.4333 with questions regarding the study
Open to Enrollment: Yes